【国泰海通医药】春节期间创新药领域重点新闻梳理-海外公司 1）2/12 公司自2019年起任职的CEO PaulHudson不再续聘，前默沙东CEO BelénGarijo上任（#过去3年赛诺菲临床失 败多管线青黄不接）； 2）2/17 赛诺菲&TEVA更新了共同开发的TL1A单抗duvakitug IIb期更长期随访的数据，其中44周时接受900mg药物维 持治疗的UC患者临床缓解率达到58%（较安 【国泰海通医药】春节期间创新药领域重点新闻梳理-海外公司 赛诺菲： 1）2/12 公司自2019年起任职的CEO PaulHudson不再续聘，前默沙东CEO BelénGarijo上任（#过去3年赛诺菲临床失 败多管线青黄不接）； 2）2/17 赛诺菲&TEVA更新了共同开发的TL1A单抗duvakitug IIb期更长期随访的数据，其中44周时接受900mg药物维 持治疗的UC患者临床缓解率达到58%（较安慰剂提高27%），CD患者的内镜应答率达到55%（较安慰剂提高 35%），相比16周均有提高，数据亮眼； 阿斯利康： 1）2/20 FDA批准阿斯利康阿克替尼联合维奈托克全口服、固定疗程方案针对一线CLL ...

Core Insights - Novartis announced positive topline results from the Phase III RemIND trial for oral remibrutinib in chronic inducible urticaria (CIndU), achieving significant complete response rates compared to placebo at Week 12 [1][2][7] - The trial met its primary endpoint across the three most prevalent types of CIndU: symptomatic dermographism, cold urticaria, and cholinergic urticaria, indicating a major advancement in treatment options for this condition [1][2][7] Company Developments - Novartis has submitted a supplemental New Drug Application (sNDA) to the FDA for remibrutinib to treat symptomatic dermographism, the most common type of CIndU [2] - The full data set from the RemIND trial will be submitted to global health authorities, and findings will be presented at upcoming medical congresses [2] Product Information - Remibrutinib is a selective oral BTK inhibitor that blocks the BTK pathway involved in histamine release, which is a key factor in hives and swelling associated with CIndU [3][7] - Currently, remibrutinib is approved in the US and China for adult patients with chronic spontaneous urticaria (CSU) who do not respond adequately to H1-antihistamines, marketed under the brand name Rhapsido [3] Clinical Trial Details - The RemIND trial is a global Phase III, multicenter, randomized, double-blind, placebo-controlled study assessing the efficacy, safety, and tolerability of remibrutinib in adults with CIndU inadequately controlled by H1-antihistamines [4] - The primary endpoint was the proportion of complete responders at Week 12, evaluated through specific provocation tests for the three CIndU subtypes [4] Market Context - CIndU affects an estimated 0.5% of the population, equating to approximately 29 million people worldwide, and is characterized by hives triggered by identifiable external factors [5][6] - There is currently a significant unmet need for effective treatment options for CIndU, as no approved targeted therapies exist [5][6]

Core Insights - Novartis (NVS) received FDA approval for Itvisma, a gene replacement therapy for spinal muscular atrophy (SMA) in patients aged two years and older, including teens and adults [1][7] - Itvisma is the first and only gene replacement therapy targeting the genetic cause of SMA, delivering a functional SMN1 gene in a single fixed dose [2][3] FDA Approval and Clinical Data - The approval was based on positive results from the phase III STEER study and the open-label phase IIIb STRENGTH study, showing significant motor function gains and stabilization of motor abilities over 52 weeks [4][7] - Itvisma demonstrated a consistent safety profile across both studies, addressing the unmet needs of approximately 9,000 individuals in the U.S. affected by SMA [5] Company Strategy and Market Position - Novartis is focusing on diversifying its portfolio amid generic competition for key drugs like Entresto, with new drug approvals and label expansions expected to mitigate adverse impacts [6] - The company’s shares have increased by 30.1% year-to-date, outperforming the industry growth of 16% [6] Future Growth Projections - Management projects a compounded annual growth rate (CAGR) of 5-6% for sales from 2025 to 2030, with an upgraded sales guidance for 2024-2029 to 6% from 5% [9] - Strong performance from key products such as Kisqali, Kesimpta, Pluvicto, and Scemblix supports the company's growth despite challenges from generic competition [10] Recent Acquisitions - Novartis announced plans to acquire Avidity Biosciences for $12 billion to enhance its late-stage neuroscience pipeline, with the acquisition expected to close in the first half of 2026 [11][12]

Financial Performance - Novartis reported a 7% increase in net sales and core operating income for Q3 2025, with a core margin of 39.3% [5][27] - Core EPS rose by 10% to $2.25, and free cash flow totaled $6.2 billion for the quarter [28][27] - For the first nine months, net sales grew by 11%, core operating income by 18%, and core EPS increased by 21% to $6.94 [28][29] Business Line Performance - Key brands showed strong growth: Kisqali grew 68%, Pluvicto 45%, Leqvio 54%, and Scemblix 95% in constant currency [6][10][14][16] - Kisqali's U.S. growth was particularly strong at 91%, with a market-leading position in both metastatic and early breast cancer [7][9] - Pluvicto's growth was driven by new patient starts, with U.S. sales up 53% [11][12] - Leqvio is on track for over $1 billion in sales, with significant growth in both the U.S. and international markets [14][15] - Scemblix is expected to become the most prescribed TKI in the U.S. [16] Market Performance - The U.S. experienced negative growth due to net true-ups related to Medicare Part D redesign, but underlying growth would have been 9% without these adjustments [27][28] - Outside the U.S., Kisqali saw 37% growth in constant currency, and Pluvicto is expanding in Europe, Canada, and Brazil [7][12] - The renal portfolio, particularly IGAN, grew 98% in the U.S., significantly outpacing market growth [20][21] Company Strategy and Industry Competition - Novartis is focused on driving growth through its priority brands and pipeline advancements, despite facing generic erosion from products like Entresto [5][28] - The company is pursuing strategic acquisitions, including Avidity Biosciences, to enhance its growth trajectory [35][36] - Novartis aims to maintain a core margin of around 40% despite expected short-term dilution from new product launches [35][36] Management Commentary on Operating Environment and Future Outlook - Management expressed confidence in mid to long-term growth, reaffirming guidance for high single-digit growth in net sales and low teens growth in core operating income for the full year [31][38] - The company anticipates challenges in the first half of 2026 due to generic competition but expects a stronger second half [33][34] - Management highlighted the importance of addressing pricing and reimbursement issues with the administration to support innovation [41][42] Other Important Information - Novartis is preparing for regulatory submissions for several products, including remibrutinib and ianalumab, with expectations for rapid uptake in the market [22][24] - The company is focused on enhancing its R&D capabilities and expanding its manufacturing base in the U.S. [43] Q&A Session Summary Question: Impact of Medicaid and tariffs on the industry - Management indicated that while some companies have reached agreements with the administration, Novartis is in ongoing discussions to find suitable solutions [41][42] Question: CapEx and cash flow modeling - Management clarified that the majority of the $23 billion commitment over five years is for R&D OpEx, with CapEx expected to remain stable at 2.5%-3% of sales [42][43] Question: Market reaction to ATR abstract - Management expressed confidence that detailed data presentation would improve market perception and emphasized the importance of patient benefits over statistical measures [46][47] Question: Pluvicto launch curve and peak sales ambition - Management stated that Pluvicto is on a strong growth trajectory, expecting to reach peak sales of $5 billion with ongoing efforts to tackle commercialization challenges [55][56][58] Question: Remibrutinib ramp-up and European adoption - Management highlighted the focus on payer negotiations and the potential for rapid uptake in the U.S. and Europe, emphasizing the innovative profile of remibrutinib [62][64] Question: Ianalumab study results and secondary endpoints - Management explained the statistical significance achieved in primary endpoints and the importance of patient-relevant outcomes for future discussions with physicians [68][71] Question: Cosentyx market share and new formulation - Management noted that there has been no significant impact from competitor launches and emphasized ongoing efforts to maintain market share in a growing market [74]

Financial Performance - In Q3 2025, net sales grew by 7% year-over-year, and core operating income also increased by 7%, with a core margin of 39.3% [4][26] - Excluding negative adjustments, underlying growth would have been 9% for net sales and 11% for core operating income, driven by priority brands and new launches [26][27] - Core EPS reached $2.25, reflecting a 10% increase, and free cash flow totaled $6.2 billion [27][28] - For the first nine months, net sales grew by 11%, core operating income increased by 18%, and core EPS was up 21% at $6.94 [27][28] Business Line Performance - Kisqali experienced a 68% growth in Q3, with U.S. growth at 91%, making it a leader in both metastatic and early breast cancer markets [5][6] - Pluvicto grew by 45% in constant currency, with U.S. sales up 53%, driven by new patient starts [10][11] - Leqvio sales increased by 54%, on track for over $1 billion in annual sales, with strong performance in both the U.S. and international markets [13][14] - Scemblix grew by 95% in constant currency, becoming the most prescribed TKI by NBRx in the U.S. [15] - Cosentyx had a mixed quarter, with a slight decline in growth due to one-time effects, but remains on track for mid-single-digit growth for the full year [16][17] Market Performance - Kisqali's early breast cancer indication is approved in 56 countries, with significant growth potential outside the U.S. [6][10] - Pluvicto is expected to reach peak sales potential with ongoing approvals in Japan and China [12] - Leqvio shows strong uptake in various international markets, particularly in China and the Middle East [14] - Scemblix is gaining traction in both first-line and second-line settings, with a strong launch momentum in Japan [15] Company Strategy and Industry Competition - The company is focused on driving growth through its priority brands while managing the impact of generic erosion from key products [4][5] - The proposed acquisition of Avidity Biosciences is expected to enhance long-term growth prospects and support mid-single-digit growth over the long term [33][34] - The company is committed to maintaining a strong pipeline and investing in R&D to support future growth [28][29] Management Commentary on Operating Environment and Future Outlook - Management expressed confidence in navigating generic headwinds and reaffirmed guidance for high single-digit growth in net sales and low teens growth in core operating income for the full year [29][30] - The company anticipates a challenging first half of 2026 due to the impact of generics but expects to emerge stronger in the second half [31][32] - Management highlighted the importance of addressing pricing and reimbursement challenges in the current regulatory environment [38][61] Other Important Information - The company achieved significant pipeline milestones, including FDA approvals and positive phase 3 results for several key products [4][20] - Management emphasized the importance of cash flow for reinvestment and shareholder returns, with a new $10 billion share buyback program announced [28][29] Q&A Session Summary Question: Industry response to Medicaid and tariffs - Management noted ongoing discussions with the administration and emphasized the need for solutions that address underlying industry issues [38] Question: CapEx over the next five years - Management clarified that the majority of the $23 billion commitment is for R&D OpEx, with no significant increase in CapEx expected [39][40] Question: Market reaction to ATR abstract - Management expressed confidence that detailed data presentation would improve market appreciation of the product [43][44] Question: Impact of Ocrevus subcutaneous launch on Cosentyx - Management indicated no significant impact from Ocrevus, with Cosentyx maintaining its market share [69][70] Question: Pluvicto launch curve and peak sales ambition - Management stated that Pluvicto is on a strong growth trajectory, with expectations of reaching $5 billion in peak sales [52][56]